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ScreenPlus: A comprehensive, multi-disorder newborn screening program

Kelly, Nicole R.; Orsini, Joseph J.; Goldenberg, Aaron J.; Mulrooney, Niamh S.; Boychuk, Natalie A.; Clarke, Megan J.; Paleologos, Katrina; Martin, Monica M.; McNeight, Hannah; Caggana, Michele; Bailey, Sean M.; Eiland, Lisa R.; Ganesh, Jaya; Kupchik, Gabriel; Lumba, Rishi; Nafday, Suhas; Stroustrup, Annemarie; Gelb, Michael H.; Wasserstein, Melissa P.
The increasing availability of novel therapies highlights the importance of screening newborns for rare genetic disorders so that they may benefit from early therapy, when it is most likely to be effective. Pilot newborn screening (NBS) studies are a way to gather objective evidence about the feasibility and utility of screening, the accuracy of screening assays, and the incidence of disease. They are also an optimal way to evaluate the complex ethical, legal and social implications (ELSI) that accompany NBS expansion for disorders. ScreenPlus is a consented pilot NBS program that aims to enroll over 100,000 infants across New York City. The initial ScreenPlus panel includes 14 disorders and uses an analyte-based, multi-tiered screening platform in an effort to enhance screening accuracy. Infants who receive an abnormal result are referred to a ScreenPlus provider for confirmatory testing, management, and therapy as needed, along with longitudinal capture of outcome data. Participation in ScreenPlus requires parental consent, which is obtained in active and passive manners. Patient-facing documents are translated into the ten most common languages spoken at our nine pilot hospitals, all of which serve diverse communities. At the time of consent, parents are invited to receive a series of online surveys to capture their opinions about specific ELSI-related topics, such as NBS policy, residual dried blood spot retention, and the types of disorders that should be on NBS panels. ScreenPlus has developed a stakeholder-based, collective funding model that includes federal support in addition to funding from 14 advocacy and industry sponsors, all of which have a particular interest in NBS for at least one of the ScreenPlus disorders. Taken together, ScreenPlus is a model, multi-sponsored pilot NBS program that will provide critical data about NBS for a broad panel of disorders, while gathering key stakeholder opinions to help guide ethically sensitive decision-making about NBS expansion.
ISSN: 2214-4269
CID: 5620602

Mitigating Risks for Racial Bias in Pulse Oximetry on Children

Verma, Sourabh; Bailey, Sean M
PMID: 37459120
ISSN: 2168-6211
CID: 5535442

Visitor restriction during the COVID-19 pandemic did not impact rates of Staphylococcus aureus colonization in the NICU patients

Evans, Hailey Zie; Bailey, Sean; Verma, Sourabh; Cicalese, Erin
OBJECTIVES/OBJECTIVE:colonization rates before and after the visitor policy change, which coincided with the exponential rise of COVID-19 cases in New York City (NYC). METHODS:colonization. RESULTS:=0.02). CONCLUSIONS:colonization rate. Hospital unit leaders may need to focus on other strategies in order to reduce colonization.
PMID: 36190160
ISSN: 1619-3997
CID: 5351352

Impact on neonatal morbidities after a change in policy to administer antenatal corticosteroids to mothers at risk for late preterm delivery

Mally, Pradeep; Katz, Julia; Verma, Sourabh; Purrier, Sheryl; Wachtel, Elena V; Trillo, Rebecca; Bhutada, Kiran; Bailey, Sean M
OBJECTIVES/OBJECTIVE:Antenatal corticosteroids (ACS) administered to mothers at risk for preterm delivery before 34 weeks has been standard care to improve neonatal outcomes. After introducing a new obstetric policy based on updated recommendations advising the administration of ACS to pregnant women at risk for late preterm (LPT) delivery (34-36 6/7 weeks), we set out to determine the short-term clinical impact on those LPT neonates. METHODS:Retrospective chart review of LPT neonates delivered at NYU Langone Medical Center both one year before and after the policy went into place. We excluded subjects born to mothers with pre-gestational diabetes, multiple gestations, and those with congenital/genetic abnormalities. We also excluded subjects whose mothers already received ACS previously in pregnancy. Subjects were divided into pre-policy and post-policy groups. Neonatal and maternal data were compared for both groups. RESULTS:388 subjects; 180 in the pre-policy and 208 in the post-policy group. This policy change resulted in a significant increase in ACS administration to mothers who delivered LPT neonates (67.3 vs. 20.6%, p<0.001). In turn, there was a significant reduction in LPT neonatal intensive care unit (NICU) admissions (44.2 vs. 54.4%, p=0.04) and need for respiratory support (27.9 vs. 42.8%, p<0.01). However, we also found an increased incidence of hypoglycemia (49.5 vs. 28.3%, p<0.001). CONCLUSIONS:This LPT ACS policy appears effective in reducing the need for LPT NICU level care overall. However, clinicians must be attentive to monitor for adverse effects like hypoglycemia, and there remains a need for better understanding of potential long-term impacts.
PMID: 36318716
ISSN: 1619-3997
CID: 5358552

Near-Infrared Spectroscopy to Guide and Understand Effects of Red Blood Cell Transfusion

Bailey, Sean M.; Mally, Pradeep V.
ISSN: 0095-5108
CID: 5568332

Near-infrared spectroscopy in the medical management of infants

Bailey, Sean M; Prakash, Shrawani Soorneela; Verma, Sourabh; Desai, Purnahamsi; Kazmi, Sadaf; Mally, Pradeep V
Near-infrared spectroscopy (NIRS) is a technology that is easy to use and can provide helpful information about organ oxygenation and perfusion by measuring regional tissue oxygen saturation (rSO2) with near-infrared light. The sensors can be placed in different anatomical locations to monitor rSO2 levels in several organs. While NIRS is not without limitations, this equipment is now becoming increasingly integrated into modern healthcare practice with the goal of achieving better outcomes for patients. It can be particularly applicable in the monitoring of pediatric patients because of their size, and especially so in infant patients. Infants are ideal for NIRS monitoring as nearly all of their vital organs lie near the skin surface which near-infrared light penetrates through. In addition, infants are a difficult population to evaluate with traditional invasive monitoring techniques that normally rely on the use of larger catheters and maintaining vascular access. Pediatric clinicians can observe rSO2 values in order to gain insight about tissue perfusion, oxygenation, and the metabolic status of their patients. In this way, NIRS can be used in a non-invasive manner to either continuously or periodically check rSO2. Because of these attributes and capabilities, NIRS can be used in various pediatric inpatient settings and on a variety of patients who require monitoring. The primary objective of this review is to provide pediatric clinicians with a general understanding of how NIRS works, to discuss how it currently is being studied and employed, and how NIRS could be increasingly used in the near future, all with a focus on infant management.
PMID: 36404215
ISSN: 1538-3199
CID: 5371942


Kilburn, L; Landi, D; Leary, S; Ziegler, D; Baxter, P; Franson, A; McCowage, G; Waanders, A; Van, der Lugt J; Yalon, Oren M; Gerber, N; Gottardo, N; Khuong-Quang, D -A; Nysom, K; Bailey, S; Driever, P H; Perreault, S; Witt, O; Hahn, S; Hargrave, D; Hassall, T; Jabado, N; Kang, H J; Larouche, V; Toledano, H; Kline, C; Abdelbaki, M; Chi, S; Gardner, S; Whipple, N; Mueller, S; Blackman, S; Zhao, X; Da, Costa D; Cox, M; Packer, R; Hansford, J
BACKGROUND: RAF alterations are oncogenic drivers found in most pediatric low-grade gliomas (LGGs). Tovorafenib is an investigational, oral, selective, CNS-penetrant, small molecule, type II pan-RAF inhibitor.
METHOD(S): FIREFLY-1 (NCT04775485) is a multicenter phase 2 study evaluating the safety and efficacy of tovorafenib monotherapy. Registrational arm 1 enrolled patients with recurrent/progressive LGG harboring an activating BRAF alteration. Patients aged 6 months-25 years who progressed following >= 1 prior line of systemic therapy were eligible. Tovorafenib 420 mg/m2 (<= 600 mg) was administered weekly (tablet or liquid suspension formulation) until progression or for >= 26, 28-day cycles. The primary endpoint (arm 1) was overall response rate, as defined by RANO criteria, per independent review.
RESULT(S): As of April 14, 2022, 25 patients were enrolled to arm 1 and had >= 6 months of follow-up. Median age at enrollment was 8 years (range 3-18). Most patients had astrocytomas (92%), 48% with optic pathway involvement. Patients were heavily pretreated (56% with >= 3 prior lines of therapy), and 72% previously received MAPK pathwaytargeted agents. Tumors harbored BRAF fusions (84%) or BRAF V600E mutations (16%). Per independent assessment, partial responses (1 unconfirmed) were seen in 14 (64%) of 22 evaluable patients, with 6 additional patients having stable disease, and a clinical benefit rate of 91%. Responses were achieved in tumors with BRAF fusions and V600E mutations. Most treatment-emergent adverse events (AEs) were grade 1 or 2 (96%). The most common grade >= 3 AEs were anemia (12%), vomiting, increased blood creatinine phosphokinase and maculopapular rash (8% each). Seven patients (28%) required dose modification for treatment-related AEs; no patients discontinued tovorafenib due to AEs. Updated results, including efficacy per RAPNO assessments will be presented.
CONCLUSION(S): Tovorafenib was generally well tolerated and showed encouraging evidence of antitumor activity in children with pretreated BRAF-altered LGG
ISSN: 1523-5866
CID: 5513272

Effects of Inhaled Iloprost for the Management of Persistent Pulmonary Hypertension of the Newborn

Verma, Sourabh; Lumba, Rishi; Kazmi, Sadaf H; Vaz, Michelle J; Prakash, Shrawani Soorneela; Bailey, Sean M; Mally, Pradeep V; Randis, Tara M
OBJECTIVE: The study aimed to evaluate the effects of inhaled iloprost on oxygenation indices in neonates with persistent pulmonary hypertension of the newborn (PPHN). STUDY DESIGN/METHODS:) were recorded. RESULTS: < 0.05), with no significant change in required mean airway pressure over that same period. There was no change in vasopressor use or clinically significant worsening of platelets count, liver, and kidney functions after initiating iloprost. CONCLUSION/CONCLUSIONS: Inhaled iloprost is well tolerated and seems to have beneficial effects in improving oxygenation indices in neonates with PPHN who do not respond to iNO. There is a need of well-designed prospective trials to further ascertain the benefits of using inhaled iloprost as an adjunct treatment in neonates with PPHN who do not respond to iNO alone. KEY POINTS/CONCLUSIONS:· Inhaled iloprost seems to have beneficial effects in improving oxygenation indices in PPHN.. · Inhaled iloprost is generally well tolerated in newborns with PPHN.. · There is a need for prospective RCTs to further ascertain the benefits of using inhaled iloprost..
PMID: 33477175
ISSN: 1098-8785
CID: 4760862

Identification and Treatment of Neonatal Seizures During Therapeutic Hypothermia and Rewarming

Verma, Sourabh; Bailey, Sean M; Mally, Pradeep V
PMID: 35377420
ISSN: 2168-6157
CID: 5201572

Outcomes of Maternal-Newborn Dyads After Maternal SARS-CoV-2

Verma, Sourabh; Bradshaw, Chanda; Auyeung, N S Freda; Lumba, Rishi; Farkas, Jonathan S; Sweeney, Nicole B; Wachtel, Elena V; Bailey, Sean M; Noor, Asif; Kunjumon, Bgee; Cicalese, Erin; Hate, Rahul; Lighter, Jennifer L; Alessi, Samantha; Schweizer, William E; Hanna, Nazeeh; Roman, Ashley S; Dreyer, Benard; Mally, Pradeep V
PMID: 32737153
ISSN: 1098-4275
CID: 4553402